Hemophilia Gene therapy is introduction of healthier gene into the body of the patient which replaces the damaged gene. Due to the progress and technology advancement Hemophilia gene therapy is soon expected to be a possibility. Hemophilia is one disease which is expected to be a target for treatment with hemophilia gene therapy. People suffering from hemophilia have difficulty and lack in forming blood clots which brings them at hug risk of uncontrolled bleeding during minor injuries and internal bleeding into muscles and joints. There are no approved Hemophilia Gene therapy presently however the treatment involve injections which clot the proteins, these proteins are expensive and cost about 1 million per year.
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Manufacturers and scientists with continuous Research and development are making efforts for successful treatment of Hemophilia by Gene therapy. Patients suffering from hemophilia A has a mutation for factor VIII in the gene and patients suffering from hemophilia B have a mutation due to the absence of clotting factor IX. The final goal of hemophilia gene therapy is the restoration of a corrected gene for the rest of the life of the patient which is a challenging and yet not accomplished. Hemophilia gene therapy treatment has a low brink for success. The hemophilia gene therapy uses viruses as a vector however the problem is the vector is not large enough which can carry all the genes and the other problem occurs during the insertion of the vector as the vector is a virus there is a possibility that it can interfere with immune system and react to it. Moreover hemophilia gene therapy is more likely to interact with the immune system if the doses are increased. The scientists and the biopharmaceutical industries are trying their best in order to cure Hemophilia A and B by gene therapy.
Tentatively, Hemophilia Gene therapy Market has been segmented on the basis of product type.
On the basis of Indication, Hemophilia Gene therapy Market can be segmented as:
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Global Hemophilia Gene therapy Market will witness advancement due to the rising population suffering from hemophilia. According to national institute of hemophilia approximately 20,000 people in U.S. and 400,000 worldwide are suffering from hemophilia. The National hemophilia foundation is awarding grants to further support the research for Hemophilia Gene therapy which will in turn help in the treatment of hemophilia. The biopharmaceutical manufacturers and research centers are working together to understand the genetics of hemophilia and improve Hemophilia Gene therapy which could help in treatment of hemophilia in the future. Manufacturers such as Roche has recently acquired Spark Therapeutics for its long term investment hemophilia A gene therapy market. Also many drugs for hemophilia gene therapy are in clinical trials. The continuous investment and research by the manufacturers is expected to improve the hemophilia gene therapy market in the coming future. Also Hemophilia Gene therapy assures to address the unmet needs by one time administration which will further improve its severity. However the arrival of Hemophilia Gene therapy is a concern over its affordability and accessibility.
Geographically, global Hemophilia Gene therapy Market is split into regions viz. North America, Latin America, Middle East & Africa, Asia Pacific, Western Europe and Eastern Europe. North America’s Hemophilia Gene therapy Market is expected to grow because of evolution and progression in the technology and advancements to improve the patients’ health. However the willingness of payers and government to arrange funding or insurance coverage for Hemophilia Gene therapy is not well established. If the manufacturers that bring Hemophilia Gene therapy to the market have conventional and older hemophilia therapies within their product portfolio their consideration to offer gene therapy for low price may lack as the new technology would disrupt their present market.
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Some of the major market members in the Global Hemophilia Gene therapy Market identified across the value chain includes: F. Hoffmann-La Roche AG, Pfizer Inc., BioMarin Pharmaceuticals, uniQure, Shire PLC, Sangamo Therapeutics, among others.
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